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Results for “GM1 Gangliosidosis, Type I”

5 trials

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Showing 5 of 5 results

Testing effectiveness (Phase 2)Looking for participantsNCT03952637
What this trial is testing

Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Who this might be right for
Lysosomal DiseasesGangliosidosisGM1
National Human Genome Research Institute (NHGRI) 54
Testing effectiveness (Phase 2)Active Not RecruitingNCT04713475
What this trial is testing

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

Who this might be right for
GM1 GangliosidosisGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2+1 more
Gemma Biotherapeutics 26
Early research (Phase 1)Not Yet RecruitingNCT07479953
What this trial is testing

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

Who this might be right for
GM1 GangliosidosesGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2
Tippi Mackenzie 5
Not applicableStudy completedNCT04041102
What this trial is testing

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Who this might be right for
GM1 Gangliosidosis
University of Pennsylvania 31
Post-approval studies (Phase 4)Ended earlyNCT02030015
What this trial is testing

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

Who this might be right for
GM1 GangliosidosesGM2 GangliosidosesTay-Sachs Disease+1 more
University of Minnesota 16